Vertex profits from Casgevy sickle cell treatment

Vertex Pharmaceuticals reported $2 million in revenue from the first patient treated with its sickle cell therapy, Casgevy. This development, alongside the commercial launch of bluebird bio’s Lyfgenia, represents a significant milestone as the first two gene therapies to potentially cure sickle cell disease gain regulatory approval. However, bluebird bio has faced considerable financial challenges, leading to cost-cutting measures and staff layoffs announced in September.

During its third-quarter earnings call, Vertex disclosed that it has collected cells from about 40 patients who are undergoing the gene-editing therapy for sickle cell disease and beta thalassemia. The company has operationalized 45 treatment centers and aims to increase this number to approximately 75 globally.

Casgevy is designed for patients with severe pain crises caused by sickle cell disease. The treatment involves extracting and gene-editing patients’ blood stem cells to express fetal hemoglobin. This is followed by an intensive conditioning regimen, which carries a high risk of infertility, before the edited cells are reintroduced into the patient’s body.

Known primarily for its cystic fibrosis treatments, Vertex announced an increase in its 2024 revenue projection from $10.8 billion to $10.9 billion.

Moreover, the biotech firm is gearing up for the potential FDA approval of suzetrigine, a new non-opioid pain medication, which is anticipated early next year. Vertex also recently commenced a Phase 3 trial named RAINIER for povetacicept, a drug targeting IgA nephropathy, which it acquired through the purchase of Alpine Immune Sciences.